Global Hematology Drugs Market Outlook, 2031

The global hematology drugs industry covers medicines for disorders of the blood and blood-forming organs, including anaemias, haemophilia and other bleeding disorders, sickle cell disease, thalassaemia, paroxysmal nocturnal haemoglobinuria, immune thrombocytopenia, myelodysplastic syndromes, myelofibrosis and other myeloproliferative neoplasms, and blood cancers such as multiple myeloma, lymphomas and leukaemias. Benign hematology products include erythropoiesis-stimulating agents, intravenous iron, clotting factor concentrates, non-factor haemophilia therapies such as Roche's Hemlibra, Novo Nordisk's Alhemo and Pfizer's Hympavzi, gene therapies such as CSL Behring's Hemgenix and Vertex and CRISPR Therapeutics' Casgevy, complement inhibitors such as AstraZeneca's Soliris and Ultomiris and Novartis's Fabhalta, thrombopoietin receptor agonists, and Bristol Myers Squibb's Reblozyl for anaemia in myelodysplastic syndromes and beta thalassaemia. Malignant hematology includes Johnson & Johnson's Darzalex and Carvykti, Bristol Myers Squibb's Revlimid, Pomalyst and Breyanzi, AbbVie and Johnson & Johnson's Imbruvica, BeiGene's Brukinsa, AstraZeneca's Calquence, AbbVie and Roche's Venclexta, Pfizer's Elrexfio and many bispecific antibodies and CAR-T therapies. Other important companies include Sanofi, Takeda, Novartis, Incyte, Amgen, Regeneron, Gilead's Kite, Geron, Agios and Sobi, while plasma product companies such as CSL, Grifols, Takeda and Octapharma supply clotting factors and immunoglobulins. Hematology has been one of the most innovative areas of medicine since 2020. Gene therapy reached patients with the approval of Hemgenix for haemophilia B in 2022, Roctavian for haemophilia A and, in December 2023, Casgevy and Bluebird Bio's Lyfgenia for sickle cell disease, with Casgevy becoming the first approved CRISPR-based therapy. Uptake has been slower than expected because of high prices of USD 2 million to more than USD 3 million, complex treatment involving chemotherapy conditioning, and limited treatment centres, and Pfizer discontinued its haemophilia B gene therapy Beqvez in 2025. In haemophilia, non-factor therapies that allow infrequent subcutaneous dosing have expanded, with the FDA approving Hympavzi in October 2024, Alhemo in December 2024 and Sanofi's Qfitlia, an siRNA therapy dosed as rarely as every two months, in March 2025. Sickle cell disease suffered a setback when Pfizer withdrew Oxbryta worldwide in September 2024 over safety concerns. Oral and new complement inhibitors changed treatment for paroxysmal nocturnal haemoglobinuria, and new drugs such as Geron's Rytelo in 2024 and Sanofi's Wayrilz for immune thrombocytopenia in 2025 addressed unmet needs. In blood cancers, bispecific antibodies and CAR-T cell therapies moved into earlier lines of treatment, and Revlimid's generic entry, which widened from 2026, reduced revenue from one of the largest hematology brands. Market Dynamics Market Drivers • High Burden of Blood Disorders and Blood CancersAnaemia affects nearly two billion people worldwide, especially women and children in low- and middle-income countries, while chronic kidney disease and cancer increase demand for anaemia treatments in older adults. Sickle cell disease affects millions of people, mainly in Africa, India and the Americas, and thalassaemia is common in the Mediterranean, Middle East and Asia. Multiple myeloma, lymphomas and leukaemias are increasing with ageing populations, and patients live longer with treatment, extending therapy duration. • Breakthrough Therapies and Precision TreatmentGene therapies, gene editing, bispecific antibodies, CAR-T cell therapies, oral complement inhibitors and siRNA drugs have transformed treatment for conditions previously managed with frequent infusions or transfusions. Many hematology drugs benefit from orphan drug designations, priority review and premium pricing. Advances in genetic testing and molecular diagnosis allow more precise targeting of therapies, supporting continued innovation and investment. Market Challenges • High Costs and Access BarriersMany new hematology therapies are among the most expensive medicines in the world, with gene therapies priced in the millions of dollars and CAR-T therapies costing several hundred thousand dollars. Payers require outcomes-based agreements, and many health systems restrict access. Most people with sickle cell disease live in sub-Saharan Africa and India, where access to advanced therapies is extremely limited, creating a large gap between innovation and global need. • Safety Risks and Complex DeliveryGene therapies require chemotherapy conditioning, which carries risks including infertility and secondary cancers, and long-term durability remains uncertain. CAR-T and bispecific therapies can cause cytokine release syndrome, neurotoxicity and infections, requiring specialist centres. The withdrawal of Oxbryta in 2024 and safety warnings for some therapies have reinforced caution. Limited treatment centre capacity and manufacturing complexity slow adoption. Market Trends • Less Frequent Dosing in Chronic Blood DisordersTreatments for haemophilia, paroxysmal nocturnal haemoglobinuria and other chronic blood disorders are moving toward infrequent subcutaneous or oral dosing. Emicizumab can be given monthly, fitusiran as rarely as every two months, and anti-TFPI antibodies weekly, while oral complement inhibitors such as iptacopan replaced infusions for some PNH patients. These options reduce treatment burden and improve adherence, although gene therapies offering one-time treatment compete with them. • Earlier Use of Bispecifics and Cell TherapiesBispecific antibodies, including teclistamab, elranatamab, talquetamab and linvoseltamab in myeloma and glofitamab, epcoritamab and mosunetuzumab in lymphoma, are moving into earlier lines of treatment and combination regimens. CAR-T therapies such as Carvykti and Breyanzi are used earlier after strong trial results. In vivo CAR-T approaches, which modify cells inside the body, and allogeneic off-the-shelf cell therapies aim to reduce cost and complexity. Segment Analysis Drugs for blood cancers account for the largest share of hematology drug value, while treatments for anaemia, bleeding disorders and rare blood diseases represent large and fast-innovating segments. Therapies for hematological malignancies, including multiple myeloma, lymphomas, leukaemias and myeloproliferative neoplasms, generate the largest share of hematology revenue. Key products include anti-CD38 antibodies such as daratumumab, immunomodulatory drugs such as lenalidomide and pomalidomide, proteasome inhibitors, BTK inhibitors such as ibrutinib, acalabrutinib, zanubrutinib and pirtobrutinib, BCL-2 inhibitors such as venetoclax, JAK inhibitors such as ruxolitinib, bispecific antibodies and CAR-T cell therapies. Anaemia treatments include erythropoiesis-stimulating agents such as epoetin and darbepoetin, HIF-PH inhibitors such as daprodustat and vadadustat, intravenous and oral iron, and luspatercept for anaemia in myelodysplastic syndromes and beta thalassaemia. Haemophilia and bleeding disorder treatments include plasma-derived and recombinant clotting factors, extended half-life factors such as Altuviiio, non-factor therapies such as emicizumab, concizumab, marstacimab and fitusiran, and gene therapies. Sickle cell disease treatments include hydroxyurea, L-glutamine, crizanlizumab and the gene therapies Casgevy and Lyfgenia. Complement inhibitors, including eculizumab, ravulizumab, iptacopan, danicopan, pegcetacoplan and crovalimab, treat PNH and related disorders. Immune thrombocytopenia is treated with thrombopoietin receptor agonists, fostamatinib and rilzabrutinib. Anticoagulant reversal agents and immunoglobulins form additional segments. Biologics and cell and gene therapies generate the largest and fastest-growing share of hematology value, while small molecules remain widely used across both benign and malignant blood disorders. Biologics, including monoclonal antibodies such as daratumumab, emicizumab, eculizumab and ravulizumab, bispecific antibodies, recombinant clotting factors and erythropoiesis-stimulating agents, account for a large share of hematology drug revenue. Bispecific T-cell engagers have been one of the fastest-growing biologic categories since 2022. Small molecules, including BTK inhibitors, BCL-2 inhibitors, immunomodulatory drugs, JAK inhibitors, oral complement inhibitors, HIF-PH inhibitors, hydroxyurea and thrombopoietin receptor agonists, are widely used and often taken orally, and several face generic competition, including lenalidomide. Cell therapies, including CAR-T therapies such as Carvykti, Abecma, Breyanzi, Yescarta, Kymriah and Tecartus, are used in myeloma, lymphoma and leukaemia and command high prices. Gene therapies and gene editing, including Casgevy, Lyfgenia, Hemgenix and Roctavian, offer potentially one-time treatments for sickle cell disease, thalassaemia and haemophilia. RNA-based therapies, including siRNA such as fitusiran, represent a newer category. Plasma-derived products, including clotting factor concentrates, immunoglobulins and albumin, are supplied by plasma fractionators. Biosimilars of epoetin, filgrastim, pegfilgrastim, rituximab and eculizumab have reduced prices in several segments. Intravenous administration accounts for a large share of hematology drug value because of infusion-based biologics and cell therapies, while subcutaneous and oral options are the fastest-growing routes. Intravenous infusion remains common for many hematology treatments, including chemotherapy, some monoclonal antibodies, CAR-T cell therapies, gene therapies, clotting factor concentrates, intravenous iron and complement inhibitors such as eculizumab and ravulizumab. These treatments are given in hospitals, specialist centres or infusion clinics, and some clotting factors are self-infused at home. Subcutaneous injection has grown rapidly, including subcutaneous daratumumab, which replaced most intravenous use, emicizumab, concizumab, marstacimab, fitusiran, crovalimab, pegcetacoplan, bispecific antibodies such as teclistamab and elranatamab, erythropoiesis-stimulating agents and pegfilgrastim. Oral administration is standard for BTK inhibitors, BCL-2 inhibitors, immunomodulatory drugs, JAK inhibitors, oral complement inhibitors, HIF-PH inhibitors, iron supplements, hydroxyurea and thrombopoietin receptor agonists, and it has expanded treatment outside hospitals. The shift from intravenous to subcutaneous and oral therapy reduces hospital visits and improves quality of life, particularly for patients with chronic disorders who need lifelong treatment. Gene therapies are given as a single intravenous infusion after cell collection and, for sickle cell disease, chemotherapy conditioning. Hospitals and specialist treatment centres are the main channel for high-value hematology therapies, while specialty and retail pharmacies distribute a growing share of oral and self-injected treatments. Hospital pharmacies supply infusion-based treatments, chemotherapy, CAR-T therapies, gene therapies and many biologics, and they are the main channel for blood cancer and severe blood disorder treatment. Specialist centres, including haemophilia treatment centres, comprehensive cancer centres and authorised cell and gene therapy centres, coordinate complex care and are required for many advanced treatments. Specialty pharmacies in the United States dispense oral oncology drugs, self-injected haemophilia therapies and complement inhibitors, managing reimbursement and patient support. Home infusion and home delivery services support patients with haemophilia who infuse clotting factors at home. Retail pharmacies dispense common products such as oral iron, hydroxyurea and some supportive medications. Blood banks and transfusion services, although not drug channels, are closely linked to anaemia and thalassaemia care. Government and tender procurement is important in Europe, China and emerging markets, where national programmes purchase clotting factors, iron and hydroxyurea. Humanitarian programmes, such as the World Federation of Hemophilia's donation programme and initiatives to expand hydroxyurea access in Africa, supply lower-income countries. Regional Analysis North America leads the hematology drugs market by value because of high prices for blood cancer drugs, early adoption of gene and cell therapies and broad access to novel treatments, while Asia-Pacific has the largest patient populations for anaemia and inherited blood disorders. North America, led by the United States, generates the largest share of hematology drug revenue. The United States is the leading market for multiple myeloma, lymphoma and leukaemia drugs, CAR-T therapies, bispecific antibodies and gene therapies, and the FDA approved many hematology products between 2023 and 2025, including Casgevy, Lyfgenia, Hympavzi, Alhemo, Qfitlia, Rytelo and Wayrilz. Medicare and Medicaid programmes, including the Centers for Medicare and Medicaid Services' Cell and Gene Therapy Access Model for sickle cell disease launched in 2025, influence access. Generic lenalidomide has reduced revenue from Revlimid. Europe is the second largest market, with strong hematology research, broad access to cancer drugs and complement inhibitors, and national agreements for gene therapies, although some gene therapies were withdrawn from Europe for commercial reasons. Asia-Pacific has large populations with anaemia, thalassaemia and haemophilia, and a growing blood cancer burden. China has become a major hematology market, with domestic companies such as BeiGene, Hengrui and Legend Biotech, which developed Carvykti with Johnson & Johnson, and expanding reimbursement. Japan has a large market for anaemia and blood cancer drugs. India has the second largest sickle cell population in the world and a national mission to eliminate the disease by 2047. Sub-Saharan Africa carries most of the global sickle cell burden but has very limited access, while the Middle East has high thalassaemia and sickle cell prevalence and is investing in advanced therapies. Key Developments August 2025: Sanofi's oral BTK inhibitor was approved for adults with persistent or chronic immune thrombocytopenia who had an insufficient response to previous treatment. March 2025: Sanofi's siRNA therapy was approved for prophylaxis in haemophilia A or B with or without inhibitors, with dosing as infrequently as every two months. February 2025: Pfizer stopped development and commercialisation of its haemophilia B gene therapy Beqvez, citing limited patient and physician interest in gene therapy. October 2024: Pfizer's anti-TFPI antibody was approved as a once-weekly subcutaneous prophylaxis for haemophilia A or B without inhibitors. September 2024: Pfizer voluntarily withdrew voxelotor for sickle cell disease from all markets after data showed an imbalance in vaso-occlusive crises and deaths. December 2023: The FDA approved Vertex and CRISPR Therapeutics' Casgevy, the first CRISPR-based therapy, and Bluebird Bio's Lyfgenia as gene therapies for sickle cell disease. Considered in this report • Historic Year: 2020 • Base Year: 2025 • Estimated Year: 2026 • Forecast Year: 2031 Aspects covered in this report • Global Hematology Drugs Market with its value and forecast along with its segments • Various drivers and challenges • Ongoing trends and developments • Top profiled companies • Strategic recommendation By Disease • Blood Cancers • Anaemia • Haemophilia and Bleeding Disorders • Sickle Cell Disease and Thalassaemia • Paroxysmal Nocturnal Haemoglobinuria • Immune Thrombocytopenia By Drug Type • Biologics • Small Molecules • Cell Therapies • Gene Therapies • Plasma-Derived Products By Route of Administration • Intravenous • Subcutaneous • Oral By Distribution Channel • Hospital Pharmacies • Specialist Treatment Centres • Specialty Pharmacies • Retail Pharmacies • Government and Tender Procurement

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Sikandar Kesari

Research Analyst

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